Immix Biopharma, Inc. announced new clinical data from its Phase 1b/2a NEXICART-1 (NCT04720313) study of novel, autologous, BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy, NXC-201, in patients with relapsed/refractory AL Amyloidosis (R/R ALA) at an oral presentation by study investigator Moshe E. Gatt, MD at the 65thAmerican Society of Hematology (ASH) Meeting being held in San Diego, CA. The updated results include follow-up and clinical data from one new patient. All patients were relapsed/refractory to standard-of-care Dara-CyBorD (daratumumab combined with cyclophosphamide, bortezomib, and dexamethasone) and had experienced a median of 6 prior lines of therapy that failed to stop worsening of disease prior to receiving NXC-201.

At the NXC-201 ASH 2023 oral presentation, data were presented from 10 relapsed/refractory AL amyloidosis patients (including one new patient) in the ongoing Phase 1b/2a NEXICART-1 study, with median 6 lines of therapy prior to NXC-201. Patients were infused with CAR+T cells at doses of 150 x 106 (n=1), 450 x 106 (n=2), and 800 x 106 (n=7).  Patient characteristics: 90% (9/10) had high-risk cytogenetics 80% (8/10) had cardiac involvement 50% (5/10) had New York Heart Association (NYHA) stage 3 or 4 heart failure (3 stage 4, 2 stage 3) 40% (4/10) had Mayo stage 3 (1 stage 3b, 3 stage 3a) AL amyloidosis disease 40% (4/10) had t(11;14) translocation Relapsed/refractory to a median 6 lines of prior therapy (range: 3-10) Safety and efficacy data: Overall response rate of 100% (10/10) Complete response + very good partial response rate of 90% (9/10) Complete response rate of 70% (7/10) (6 out of 7 were MRD 10-5) Organ response rate of 60% (6/10) Best responder had a duration of response of 23.7 months as of December 10, 2023, with response ongoing Transformation to complete response at month 2 observed in a patient with 7 lines of prior therapy and cardiac involvement There were no immune effector cell-associated neurotoxicity syndrome (ICANS) events ?Single Day CRS?: Median CRS duration was 1 day (range: 1-4): No grade 4 cytokine release syndrome (CRS) events 2 experienced no CRS; 2 experienced grade 1 CRS; 4 Experienced grade 2 CRS; 2 experienced grade 3 CRS For the 8 patients with cardiac involvement: Overall response rate of 100% (8/8) Complete response rate of 63% (5/8) (4 out of 5 were MRD 10-5) Organ response rate of 63% (5/8) For the 4 patients with t(11;14) disease: Overall response rate of 100% (4/4) Complete response rate of 75% (3/4) (MRD 10-5) Organ response rate of 50% (2/4).